• Mary Skrypek, Nicholas Foreman, Daniel Guillaume, and Christopher Moertel.
• Department of Pediatric Hematology and Oncology, University of Minnesota, Minneapolis, Minnesota.
• Pediatr Blood Cancer. 2014 Nov 1;61(11):2099-100.

AbstractThe BRAF V600E missense mutation is known to be present in a subset of central nervous system tumors. We report a patient with a BRAF V600E mutated pilomyxoid astrocytoma who failed multiple conventional chemotherapy regimens. Treatment with vemurafenib, a molecularly targeted therapy against the mutant BRAF V600E kinase, combined with vinblastine resulted in tumor regression. Furthermore, this patient experienced almost immediate progression of disease after holding vemurafenib for only 2-3 weeks, suggesting that the tumor response is vemurafenib dependent. This population of patients may benefit from targeted therapy and testing of individual tumors for BRAF mutations is justified.© 2014 Wiley Periodicals, Inc.

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